The BioPharm Brief: Survival, Gene Editing, and Skin Cancer

Welcome to The BioPharm Brief, your daily snapshot of developments shaping the biopharmaceutical industry. Here’s what’s making headlines today.

First, Roche’s B7-H3-targeted antibody-drug conjugate Tam-Peli has delivered positive phase 3 results in relapsed small-cell lung cancer. In the TAISHAN-302 trial, Tam-Peli reduced the risk of death by 54% compared with topotecan. Median overall survival was 13.3 months versus 9.4 months, while progression-free survival was 7.4 months versus 2.8 months. The 451-patient study also showed a response rate of 59.1% with Tam-Peli compared with 9.7% for topotecan. Roche plans to move the program into global phase 3 development.

Cellectis is shifting its focus to in vivo gene editing after exiting its allogeneic CAR T programs. The company will prioritize .HEAL-101, which targets APOC3 for severe hypertriglyceridemia, and .HEAL-201, which targets PCSK9 for severe hypercholesterolemia. Both programs use lipid nanoparticles to deliver TALE-based editors, with the company targeting first phase 1 readouts in 2027 and 2028. Preclinical studies showed roughly 76% triglyceride reduction with .HEAL-101 and more than 90% plasma PCSK9 reduction with .HEAL-201.

And Phio Pharmaceuticals has submitted an FDA briefing package to support a planned phase 2b trial of PH-762 in cutaneous squamous cell carcinoma. In its phase 1b study, PH-762 produced a 70% overall response rate in squamous cell carcinomas, with complete clearance reported in 10 of 14 responders. The intratumoral siRNA therapy is designed to silence PD-1 using Phio’s INTASYL platform, with clinical supplies for the planned phase 2b study expected in the first quarter of 2027.

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Key takeaways:

• Roche: Tam-Peli cut the risk of death by 54% in relapsed SCLC.
• Cellectis: In vivo gene editing becomes the company’s new priority.
• Phio: PH-762 moves toward phase 2b after a 70% response rate in phase 1b.